Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

被引：65
|
作者：
Chinen, Javier
Davis, Joie
De Ravin, Suk See
Hay, Beverly N.
Hsu, Amy P.
Linton, Gilda F.
Naumann, Nora
Nomicos, Effie Y. H.
Silvin, Christopher
Ulrick, Jean
Whiting-Theobald, Narda L.
Malech, Harry L.
Puck, Jennifer M.
机构：
[1] NIAID, Host Def Lab, NIH, Dept Hlth & Human Serv, Bethesda, MD 20892 USA
[2] NHGRI, Genet & Mol Biol Branch, NIAID, NIH,DHHS, Bethesda, MD 20892 USA
来源：
BLOOD | 2007年 / 110卷 / 01期
关键词：
BONE-MARROW-TRANSPLANTATION; CHRONIC GRANULOMATOUS-DISEASE; STEM-CELL TRANSPLANTATION; T-CELL; CD34(+) CELLS; TRANSDUCTION; SCID-X1; SURVIVAL; MODEL; IL2RG;
D O I：
10.1182/blood-2006-11-058933
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (gamma c) of receptors for interleukins 2 (IL-2), -4, -7, -9, -15, and -21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34(+) hematopoletic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.
引用
收藏
页码：67 / 73
页数：7
相关论文
共 50 条
  • [1] The evolution of gene therapy in X-linked severe combined immunodeficiency
    Rans, Tonya S.
    England, Ronald
    ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY, 2009, 102 (05) : 357 - 363
  • [2] Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
    Hacein-Bey-Abina, Salima
    Hauer, Julia
    Lim, Annick
    Picard, Capucine
    Wang, Gary P.
    Berry, Charles C.
    Martinache, Chantal
    Rieux-Laucat, Frederic
    Latour, Sylvain
    Belohradsky, Bernd H.
    Leiva, Lily
    Sorensen, Ricardo
    Debre, Marianne
    Casanova, Jean Laurent
    Blanche, Stephane
    Durandy, Anne
    Bushman, Frederic D.
    Fischer, Alain
    Cavazzana-Calvo, Marina
    NEW ENGLAND JOURNAL OF MEDICINE, 2010, 363 (04) : 355 - 364
  • [3] Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease
    Blanco, Elena
    Izotova, Natalia
    Booth, Claire
    Thrasher, Adrian James
    FRONTIERS IN IMMUNOLOGY, 2020, 11
  • [4] Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency
    De Ravin, Suk See
    Wu, Xiaolin
    Moir, Susan
    Anaya-O'Brien, Sandra
    Kwatemaa, Nana
    Littel, Patricia
    Theobald, Narda
    Choi, Uimook
    Su, Ling
    Marquesen, Martha
    Hilligoss, Dianne
    Lee, Janet
    Buckner, Clarissa M.
    Zarember, Kol A.
    O'Connor, Geraldine
    McVicar, Daniel
    Kuhns, Douglas
    Throm, Robert E.
    Zhou, Sheng
    Notarangelo, Luigi D.
    Hanson, I. Celine
    Cowan, Mort J.
    Kang, Elizabeth
    Hadigan, Coleen
    Meagher, Michael
    Gray, John T.
    Sorrentino, Brian P.
    Malech, Harry L.
    SCIENCE TRANSLATIONAL MEDICINE, 2016, 8 (335)
  • [5] Gene therapy for X-linked severe combined immunodeficiency: Historical outcomes and current status
    Pai, Sung-Yun
    Thrasher, Adrian J.
    JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2020, 146 (02) : 258 - 261
  • [6] Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
    Cavazzana, Marina
    Six, Emmanuelle
    Lagresle-Peyrou, Chantal
    Andre-Schmutz, Isabelle
    Hacein-Bey-Abina, Salima
    HUMAN GENE THERAPY, 2016, 27 (02) : 108 - 116
  • [7] Canine X-linked severe combined immunodeficiency
    Felsburg, PJ
    Somberg, RL
    Hartnett, BJ
    Henthorn, PS
    Carding, SR
    IMMUNOLOGIC RESEARCH, 1998, 17 (1-2) : 63 - 73
  • [8] Gene Therapy Model of X-linked Severe Combined Immunodeficiency Using a Modified Foamy Virus Vector
    Horino, Satoshi
    Uchiyama, Toru
    So, Takanori
    Nagashima, Hiroyuki
    Sun, Shu-lan
    Sato, Miki
    Asao, Atsuko
    Haji, Yoichi
    Sasahara, Yoji
    Candotti, Fabio
    Tsuchiya, Shigeru
    Kure, Shigeo
    Sugamura, Kazuo
    Ishii, Naoto
    PLOS ONE, 2013, 8 (08):
  • [9] A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
    Hacein-Bey-Abina, S.
    Pai, S. -Y.
    Gaspar, H. B.
    Armant, M.
    Berry, C. C.
    Blanche, S.
    Bleesing, J.
    Blondeau, J.
    de Boer, H.
    Buckland, K. F.
    Caccavelli, L.
    Cros, G.
    De Oliveira, S.
    Fernandez, K. S.
    Guo, D.
    Harris, C. E.
    Hopkins, G.
    Lehmann, L. E.
    Lim, A.
    London, W. B.
    van der Loo, J. C. M.
    Malani, N.
    Male, F.
    Malik, P.
    Marinovic, M. A.
    McNicol, A. -M.
    Moshous, D.
    Neven, B.
    Oleastro, M.
    Picard, C.
    Ritz, J.
    Rivat, C.
    Schambach, A.
    Shaw, K. L.
    Sherman, E. A.
    Silberstein, L. E.
    Six, E.
    Touzot, F.
    Tsytsykova, A.
    Xu-Bayford, J.
    Baum, C.
    Bushman, F. D.
    Fischer, A.
    Kohn, D. B.
    Filipovich, A. H.
    Notarangelo, L. D.
    Cavazzana, M.
    Williams, D. A.
    Thrasher, A. J.
    NEW ENGLAND JOURNAL OF MEDICINE, 2014, 371 (15) : 1407 - 1417
  • [10] Review: oncogenic Insertional Mutagenesis as a Consequence of Retroviral Gene Therapy for X-Linked Severe Combined Immunodeficiency Disease
    Ahmed, Bilal
    Zafar, Maria
    Qadir, Muhammad Imran
    CRITICAL REVIEWS IN EUKARYOTIC GENE EXPRESSION, 2019, 29 (06): : 511 - 520
12345